RNA is a very special kind of drug. It offers potent, specific, and precise effects in lots of diseases. To realize its therapeutic talent, RNA must act inside chosen cells. Since RNA itself cannot travel in the body, we load it into nanoparticles or other carriers, which improve RNA delivery into cells of interest. To achieve such a precise addressing, we use “ligands”, i.e., molecules with affinity to specific binding sites or “epitopes” on surface of target cells.
Carrier geometry (size and shape), biomechanics (rigidity vs flexibility), mode of ligand attachment, all modulate targeting in a complex unpredictable way. Next, distinct ligands binding to the same epitope, may differently affect host defense or blood clearance. In some cases, ligand is not needed: carriers encounter countless blood components, some of which may serve as endogenous ligands. Further, carrier binding to some epitopes implicated in the internalization favors the ultimate addressing – interior compartments of the target cell.
Our mission is to support advancement of RNA biotheraptuics via enabling swift and low-failure solutions of targeting-related aspects – rationally designed and encompassed by chance by the Institute investigators. The Core’s resources – unique wealth of methodologies, equipment, reagents, and first of all, strong and friendly team of accomplished researchers immersed in collaborative projects on targeted drug delivery for several decades, will provide comprehensive support in identification of epitopes and ligands, molecular design and testing of the targeting RNA to diverse multitudes of desirable addresses in the body.